Effective Small Interfering RNA Therapy to Treat CLCN7-dependent Autosomal Dominant Osteopetrosis Type 2
In about 70% of patients affected by autosomal dominant osteopetrosis type 2 (ADO2), osteoclast activity is reduced by heterozygous mutations of the CLCN7 gene, encoding the ClC-7 chloride/hydrogen antiporter. CLCN7G215R-, CLCN7R767W-, and CLCN7R286W-specific siRNAs silenced transfected mutant mRNA/...
Main Authors: | , , , , |
---|---|
Format: | Online |
Language: | English |
Published: |
Nature Publishing Group
2015
|
Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4877447/ |